ProQR Therapeutics Spins Out CNS-Focused Biotech, Amylon Therapeutics

Published: Sep 12, 2017

ProQR Therapeutics Spins Out CNS-Focused Biotech, Amylon Therapeutics September 12, 2017
By Mark Terry, Breaking News Staff

Leiden, the Netherlands – ProQR Therapeutics has spun out Amylon Therapeutics to focus on central nervous system disorders.

ProQR gave Amylon an exclusive license to develop drugs for beta amyloid-related disorders, with an initial focus on Hereditary Cerebral Hemorrhage with Amyloidosis of the Dutch type (HCHWA-D). This is a rare genetic disorder that causes dementia and strokes at mid-adulthood. Another future beta amyloid-related disorder the company may pursue is cerebral amyloid angiopathy (CAA).

ProQR was founded in 2012 and focuses on innovative RNA therapies for severe and rare genetic diseases. Its deep pipeline was spread across separate business units that focused on cystic fibrosis, eye diseases, skin diseases, and CNS diseases.

“As part of our corporate strategy to bring as many of our programs to patients as possible, we built ProQR as a group of focused business units that enable us to attract external funding directly into the individual development programs or ultimately spin programs out as we have now done with Amylon,” said Daniel de Boer, ProQR’s chief executive officer, in a statement. “Through spinning out Amylon, we position the beta amyloid program for optimal success with a dedicated team and the funding to develop treatments for patients suffering from these devastating brain diseases. Through our involvement in the Supervisory board and the scientific advisory board of Amylon we will continue to assist Thomas and his team in building success.”

Thomas de Vlaam will be Amylon’s founding chief executive officer. He joined ProQR in 2015 to develop the company’s CNS operations with the goal of a spinout. Dinko Valerio, chairman of the supervisory board of ProQR, will join Amylon’s supervisory board. Gerard Platenburg, ProQR’s chief innovation officer, will join Amylon’s scientific advisory board.

ProQR will retain a majority ownership in Amylon, and is entitled to milestones and royalties from any products developed by the company. Amylon also received seed funding from an unidentified group of institutional and private investors.

Although there is no indication it will focus on Alzheimer’s disease, Alzheimer’s is the best-known disease related to amyloid-beta accumulation. Thomas Wisniewski, director of the Alzheimer’s Disease Research Center in New York, who will join Amylon’s scientific advisory board, said in a statement, “The unmet need of patients living with beta amyloid related disorders is very significant and to date no disease modifying treatments are available. The technology that Amylon is developing for these patients is built on a solid scientific rationale and holds great promise for patients.”

In addition to Wisniewski and Platenburg, Philip Scheltens, professor of Neurology and director of Alzheimer Center at the VU University Medical Center Amsterdam will also serve on the scientific advisory board.

On Sept. 5, ProQR announced that its drug candidate QRX-421 for Usher Syndrome received Orphan Drug Designation from both the .S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). And on August 29, it announced that it had completed dosing of cystic fibrosis patients in its Phase Ib clinical trial of QR-010, and topline data was expected on Monday, Sept. 25, 2017.

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